New targeted pill shows promise for rare blood cancer

NCT ID NCT03580655

Completed Disease control Sponsor: Blueprint Medicines Corporation Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 2 study tested avapritinib, a daily tablet that targets a specific genetic mutation, in 107 people with advanced systemic mastocytosis (a rare blood cancer). The main goal was to see how many patients had their tumors shrink or symptoms improve. Results suggest the drug may help control the disease and ease symptoms, but more research is needed.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: avapritinib (a targeted cancer drug taken as a tablet)
What this could lead to: If successful, this could provide a new treatment option that shrinks tumors and improves symptoms for people with advanced systemic mastocytosis.
What could go wrong: This is a single-arm Phase 2 study with no comparison group, so results may not hold up in larger trials. The drug may cause side effects and not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

aggressive systemic mastocytosis mast cell leukemia Mastocytosis, Systemic systemic mastocytosis with an associated clonal hematologic non-mast cell lineage disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ADVANCED SYSTEMIC MASTOCYTOSIS AGGRESSIVE SYSTEMIC MASTOCYTOSIS MAST CELL LEUKEMIA SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGIC NEOPLASM

Contacts and locations

Locations

A.O. OO.RR. S.Giovanni di Dio e Ruggi d'Aragona, University of Salerno

Salerno, 84131, Italy
Azienda Ospedaliero-Universitaria Careggi, CRIMM - Centro di Ricerca ed Innovazione per le Malattie Mieloproliferative

Florence, 50134, Italy
Beatson West of Scotland Cancer Centre - NHS Greater Glasgow and Clyde

Glasgow, G12 0YN, United Kingdom
CHU Toulouse - Hôpital Larrey

Toulouse, 31059, France
Centro Ricerche Cliniche di Verona - Azienda Ospedaliera Universitaria Integrata di Verona

Verona, 37134, Italy
Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital

London, SE1 9RT, United Kingdom
Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
Huntsman Cancer Institute

Salt Lake City, Utah, 84112, United States
Hôpital Necker-Enfants Malades

Paris, 75015, France
Klinik und Poliklinik für Innere Medizin III, Klinikum rechts der Isar der TU München

Munich, 81675, Germany
Mays Cancer Center

San Antonio, Texas, 78229, United States
Medizinische Universität Wien, Universitätsklinik für Innere Medizin I, Klinische Abteilung für Hämatologie und Hämostaseologie

Vienna, 1090, Austria
Odense University Hospital, Department of Haematology

Odense, DK-5000, Denmark
Oslo University Hospital-Rikshospitalet, Hematology

Oslo, 0372, Norway
Roswell Park Comprehensive Cancer Center

Buffalo, New York, 14263, United States
Rush University Medical Center

Chicago, Illinois, 60612, United States
St. Michael's Hospital

Toronto, Ontario, M5B 1W8, Canada
Stanford Cancer Institute

Stanford, California, 94305, United States
The University of Kansas Cancer Center

Westwood, Kansas, 66205, United States
Uniklinik RWTH Aachen, Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltranslplantation

Aachen, 52074, Germany
University Medical Center Groningen (UMCG)

Groningen, 9713 GZ, Netherlands
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Pennsylvania, Abramson Cancer Center

Philadelphia, Pennsylvania, 19104, United States
University of Texas, MD Anderson Cancer Center

Houston, Texas, 77030, United States
Universitätsklinikum Hamburg-Eppendorf, Zentrum für Onkologie

Hamburg, 20246, Germany
Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I - Hämatologie und Zelltherapie, lnternistische Onkologie, Hämostaseologie

Leipzig, 04103, Germany
Universitätsmedizin Mannheim III. Medizinische Klinik

Mannheim, 68167, Germany
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu, Klinice Hematologii, Nowotworów Krwi i Transplantacji Szpiku

Wroclaw, 50-367, Poland
Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii

Gdansk, 80-214, Poland
Washington University School of Medicine

St Louis, Missouri, 63110, United States
lnstituto de Estudios de Mastocitosis de Castilla la Mancha, Hospital Virgen del Valle - Complejo Hospitalario de Toledo

Toledo, 45071, Spain

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